RecruitingNot ApplicableNCT07183878

Venetoclax-Enhanced BUCY vs. Standard BUCY Conditioning in High-Risk AML and MDS Patients Undergoing Allo-HSCT (Ven-BUCY Study)

A Prospective, Multicenter, Randomized Controlled Study Comparing Venetoclax-Enhanced BUCY With Standard BUCY Conditioning in High-Risk AML and MDS Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Sponsor

First Affiliated Hospital of Zhejiang University

Enrollment

138 participants

Start Date

Aug 20, 2025

Study Type

INTERVENTIONAL

Conditions

Acute Myeloid Leukemia Myelodysplastic Syndromes High-Risk Acute Myeloid Leukemia High-Risk Myelodysplastic Syndromes

Summary

This is a prospective, multicenter, randomized controlled trial designed to evaluate the efficacy and safety of venetoclax-enhanced BUCY (Ven-BUCY) conditioning compared to the standard BUCY regimen in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible participants aged 12 to 60 years will be randomized 1:1 to receive either Ven-BUCY or standard BUCY conditioning. The primary endpoint is relapse-free survival (RFS) at two years post-transplant. Secondary outcomes include overall survival, relapse rate, non-relapse mortality, measurable residual disease (MRD), and treatment-related adverse events. The study aims to improve post-transplant outcomes by deepening disease remission through the addition of venetoclax, a BCL-2 inhibitor known to target leukemia stem cells and enhance chemotherapy sensitivity.

Eligibility

Min Age: 12 YearsMax Age: 60 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a new stem cell transplant preparation approach for people with high-risk leukemia (AML) or myelodysplastic syndrome (MDS). It adds venetoclax, a targeted cancer drug, to the standard chemotherapy conditioning regimen used before transplant. **You may be eligible if...** - You are aged 12–60 with a diagnosis of high-risk AML or MDS - You have achieved remission before the transplant (or have less than 20% bone marrow blasts if you have MDS) - A matched donor (related or unrelated) is available - Your heart, liver, and kidney function meet required levels **You may NOT be eligible if...** - You have uncontrolled heart disease or severe heart failure - You have HIV or uncontrolled hepatitis B or C - You have previously had more than one stem cell transplant - You have had a severe reaction (grade 3 or higher) to venetoclax before - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVenetoclax

Venetoclax is administered orally at 400 mg/day for participants ≥14 years or 360 mg/m²/day for those aged 12-14 years, from day -14 to -8 before allogeneic hematopoietic stem cell transplantation (allo-HSCT). Dose is adjusted to 100 mg/day (or 90 mg/m²/day for pediatric patients) if used with strong CYP3A4 inhibitors such as posaconazole.

OTHERNone-placebo

Participants in this arm will not receive venetoclax as part of their conditioning regimen. They will undergo standard myeloablative conditioning with BUCY (busulfan, cyclophosphamide, and MeCCNU), prior to allogeneic hematopoietic stem cell transplantation. This arm serves as the active comparator to evaluate the addition of venetoclax in the experimental arm.